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Sirolimus+Ruxolitinib+Mycophenolate Mofetil for Prophylaxis of aGVHD in Patients Receiving Haplo-HSCT Who Are Intolerant to CNI

ConditionsAcute LeukemiaMyelodysplastic SyndromesSevere Aplastic Anemia (SAA)
Trial statusNot yet recruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age14+
SponsorPeking University People's Hospital

About this trial

Graft-versus-host disease (GVHD) is an important complication after transplantation, with an incidence of 40-60%, which can increase non-relapse mortality if poorly controlled. At present, the standard prophylaxis for GVHD is cyclosporine combined with methotrexate. However, calcineurin inhibitors (CNI) can cause some vital side effects, which are not tolerated by some patients. Therefore, this study aims to explore the safety and efficacy of Sirolimus in combination with Ruxolitinib and Mycophenolate Mofetil for the prophylaxis of GVHD in patients with haplo-HSCT who are intolerant to calcineurin inhibitors.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Primary disease: hematological malignancies (including acute leukemia, myelodysplastic syndromes), nonmalignant disorders (including severe aplastic anaemia)

Renal injury or inability to tolerate the side effects of CNI: such as CNI renal toxicity (creatinine levels above the upper limit of normal), uncontrolled hypertension, and neurotoxicity rrom the time of hematopoietic stem cell infusion until +90 days after transplantation

Receiving haplo-HSCT for the first time

Disqualifiers

Allergy or intolerance to study drugs

Active infection

Active GVHD

Transplantation-associated thrombotic microangiopathy

Trial design

Design model

Single group

Treatments tested in this trial

  • Sirolimus

    Drug

    Sirolimus 2mg once daily, maintaining the concentration at 5-10 ng/ml. Gradually reduce the dosage after +100 days. If the patient has stable engraftment and no GVHD, discontinue on +180 days.

  • Ruxolitinib

    Drug

    Ruxolitinib is administered at a dose of 5mg twice daily from the start of the study until +90 days. The dose is reduced to 5mg once daily on +90 days, and discontinued on +120 days.

  • MMF

    Drug

    MMF 0.5g, taken twice daily, is discontinued after 60 days. If it is resumed after 60 days, it should be taken for 2 weeks.

  • ATG

    Drug

    2.5 mg/kg, from -5d to -2d

Treatment groups

40 Participants
are divided into 1 treatment group
Group A: Sirolimus+Ruxolitinib+Mycophenolate mofetil (MMF)+anti-thymocyte globulin (ATG)Experimental treatment 4 interventions

Trial outcomes

Primary outcomes

1

Incidence of Grade 2-4 aGVHD within 100 days post transplantation

Time frame
Participants will be followed for an expected average of 100 days post transplantation

Secondary outcomes

1

Incidence of chronic GVHD (cGVHD) within 1 year post transplantation

Time frame
Participants will be followed for an expected average of 1 year
2

Incidence of thrombotic microangiopathy within 1 year post transplantation

Time frame
Participants will be followed for an expected average of 1 year
3

Cumulative incidence of relapse

Time frame
Participants will be followed for an expected average of 1 year
4

Transplant-related mortality

Time frame
Participants will be followed for an expected average of 1 year

Other outcomes

Locations

This trial has no locations.

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Peking University People's Hospital

Lead sponsor

Source ClinicalTrials.gov

This trial is not recruiting at the moment. You can still explore other options: